Pegcetacoplan superior to eculizumab for paroxysmal nocturnal hemoglobinuria: NEJM
Delhi: Pegcetacoplan provides broad hemolysis control and is superior to eculizumab for improving hemoglobin and clinical outcomes in paroxysmal nocturnal hemoglobinuria (PNH) patients, finds a recent study. The findings of the study are published in the New England Journal of Medicine.
Paroxysmal nocturnal hemoglobinuria is a rare blood disease characterized by chronic complement-mediated hemolysis. Intravascular hemolysis can be controlled by C5 inhibition in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan is a pegylated peptide that targets proximal complement protein C3 and potentially inhibits both intravascular and extravascular hemolysis.
The researchers conducted a phase 3 open-label, controlled trial that was aimed to assess the safety and efficacy of pegcetacoplan as compared with eculizumab in adults with PNH and hemoglobin levels lower than 10.5 g per deciliter despite eculizumab therapy. After a 4-week run-in phase in which all patients received pegcetacoplan plus eculizumab, patients were randomly assigned to subcutaneous pegcetacoplan monotherapy (41 patients) or intravenous eculizumab (39 patients).
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